Gene editing could cure cystic fibrosis before birth, study suggests

Scientists may soon be able to gene-edit horrible lung diseases like cystic fibrosis out of the human genome, a new study in mice suggests. 

The CRISPR gene editing tool allows scientists to effectively 'cut and paste' bits of DNA. 

It has been hailed as one of the greatest scientific advancements of the last several decades, and the medical community is looking to it as the best hope for treating otherwise incurable inherited diseases. 

Now, University of Pennsylvania researches have successfully used CRISPR to fix DNA defects that cause many lung diseases in mice embryos. 

The new study suggests we might someday be able to cure genetic diseases before birth in humans, too - but changing unborn babies may cause ethical controversy.

Scientists successfully edited the faulty DNA that causes lung diseases like cystic fibrosis in mice, suggesting that the CRISPR tool might someday eliminate the illness from humans

Scientists successfully edited the faulty DNA that causes lung diseases like cystic fibrosis in mice, suggesting that the CRISPR tool might someday eliminate the illness from humans

About 1,000 people will be diagnosed with the common genetic lung disease, cystic fibrosis, in the US this year. 

The majority of them will be under two, and few will survive to see their 40th birthdays. 

Broadly, respiratory problems account for over 20 percent of hospital admissions among children. 

Cystic fibrosis is devastating, causing mucous to build up in the lungs and eventually making it impossible to breathe, but some related genetic faults prove deadly before or immediately after birth.  

In addition to cystic fibrosis, surfactant protein syndromes and alpha-1 antitrypsin deficiency are each caused by errors in a single gene.  

That single mistake has lethal results, but this also makes these diseases good targets for CRISPR editing. 

The CRISPR can be tweaked to target these specific genes, snip them out and replace them with correct versions. 

But getting it right requires impeccable timing, so that the gene DNA change will affect the development of the lungs' lining, but not create broader ripple effects than clinicians

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